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Zachary Roberts, EVP of R&D and CMO, expressed confidence that MRD conversion is a strong correlate for long-term disease control based on growing data in the field. He confirmed MRD conversion data would be shared at the interim analysis but did not commit to other metrics. For ALLO-329, he stated a 'meaningful amount of data' would be shared in H1 2026 but did not provide specific patient numbers.

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President and CEO Dr. David Chang expressed confidence, explaining that the FDA is an institution that follows established guidance. He differentiated autoimmune trials from oncology, noting their endpoints are based on symptom improvement, not overall survival, which has been a focus of Dr. Prasad. He sees no new hurdles for advancing CAR T in autoimmune disease, provided the safety and efficacy data are strong.

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President and CEO Dr. David Chang clarified that it is a standard futility analysis assessing the totality of data. The primary efficacy metric will be the MRD conversion rate, with the expectation that the treatment arms will show a significantly higher rate of conversion from MRD-positive to MRD-negative compared to the observation arm. Safety will also be a key component.

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CEO Sean Nolan detailed the regulatory progress, noting that Health Canada issued a 'no objection letter,' clearing the trial to proceed. For the FDA, he confirmed receipt of correspondence indicating no clinical hold concerns, allowing them to proceed at their discretion. He characterized ongoing FDA dialogue as constructive and focused on refining program details for success. Additionally, he mentioned a scientific advice meeting with European regulators is scheduled for the early fall.

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CEO Sean Nolan assured that the core FDA review team has remained consistent and that interactions after Dr. Peter Marks' departure were unaffected, with meetings proceeding on schedule. President and Head of R&D Sukumar Nagendran added that the team reviewing the Rett program has been stable, expressing confidence in the agency's data-driven approach under new leadership.

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CEO Sean Nolan clarified that Taysha has five active dosing sites for the REVEAL trials. He confirmed that patients in the high-dose cohort have been treated at a minimum of two different sites, and across the entire program (low and high doses), at least three distinct sites have now administered the therapy.

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President and Head of R&D, Sukumar Nagendran, noted that high-dose patients will have baseline CGIS scores in the 4-6 range per protocol and exhibit common Rett syndrome features. CEO Sean Nolan added that the expectation for low-dose patients is to see continued progress, similar to the first patient who showed ongoing improvements in motor function and communication. He expressed hope for potentially better progress in the younger pediatric patients over time.

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David Berman, EVP of R&D, stated the trial is modeled on a historical one-year survival of 55% for the control arm. He confirmed that dose escalation for both PWELL and the PRAME half-life extension (HLE) should complete in the next 12 months, making it possible that HLE data could also be shared next year.

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CEO Bahija Jallal confirmed that the interim data will not be released publicly to maintain trial integrity. David Berman, Head of R&D, added that there is no futility analysis planned. The IDMC will use both efficacy and safety data to select a dose, looking for large differences between the two active doses and using modeling for support if needed.

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Mohammed Dar, an executive, provided updates on program progress, including PIWIL site activation and HLE PRAME submission, but stated it was too early to guide on data timelines for these first-in-human trials. David Berman, Head of R&D, added that learnings from the current platform are being applied to accelerate these new trials.

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Mohammed Dar, an executive, stated that for the PIWIL program, they aim to enroll the first patient by year-end. The Half-Life Extended PRAME has been submitted for regulatory feedback, and the A24 version is on track for submission by year-end. He noted it's too early to guide on data timelines. David Berman, Head of R&D, added that learnings from existing programs are being applied to accelerate these Phase I trials.

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Emil Kakkis, Founder, President & CEO, did not provide the standard deviation but acknowledged significant variation. He expressed confidence in the negative binomial model used in the SAP, noting it's the best method for fracture data and allows for co-variables to manage variation. He also stated there are no specific plans yet to present updated Phase II data.

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CEO Emil Kakkis explained that due to a 'hockey stick' enrollment pattern, the vast majority of patients had less than one year of data at the first interim. CMO Eric Crombez added that while they are formally tracking pain, they have heard significant anecdotal reports of pain improvement from the Phase II study. Kakkis noted that, similar to Crysvita, patients feeling better and becoming more active is a powerful, tangible benefit that drives enthusiasm for the therapy.

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CEO Emil Kakkis clarified that the interim analyses are now set on a fixed timeline (year-end/early 2025 and mid-2025). He explained this was an operational choice to simplify planning, as the original event-based trigger was challenging. The timeline was set by estimating when fracture event milestones would be hit, and they are confident the actual fracture rate will be sufficient by those dates.

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President and CEO Dr. Jennifer Buell stated that the NAIAD funding timeline is fluid but expressed optimism, noting the trial is submitted and ready to proceed once funding is cleared. She mentioned backup strategies include partner interest and targeted financing. Dr. Buell also confirmed that the company's current cash position provides a runway through the end of 2025.

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